Selected Medicines in Development for Neurological Disorders

Glioblastoma – Brain cancers are among the most difficult cancer to treat. According to the American Brain Tumor Association, about 45,000 primary brain tumors, both malignant and benign, will be diagnosed in the U.S. this year. About 20 percent of those will be glioblastomas, a highly invasive form of cancer that sends cancerous cells throughout the brain and spinal cord. A potential weapon against this disease is a new drug that singles out and destroys the malignant cells without harming the healthy cells

Epilepsy – A seizure disorder affecting more than 3 million Americans of all ages, according to the Epilepsy Foundation. A potential medicine in development activates ion channels in the brain that are important in the regulation of the resting potential and electrical firing of certain nerve cells. In clinical trials the medicine has shown activity against partial seizures, generalized seizures and treatment-resistant seizures.

Fibromyalgia – A chronic pain syndrome, affects approximately 2 percent of the U.S. population, of which most are women, according to the Arthritis Foundation. Symptoms can be debilitating and are characterized by chronic and widespread pain throughout the body, often accompanied by severe fatigue and poor sleep. One medicine in development is the first in a new class of drugs known as norepinephrine serotonin reuptake inhibitors. The medicine decreases the uptake of two key neurotransmitters – norepinephrine and serotonin, which play an essential role in regulating pain and mood.

Migraine – A painful neurologic condition that affects more than 29.5 million Americans, with women being affected three times more often than men, according to the National Headache Foundation. One first-in-class medicine in development selectively blocks transmission of pain signals to the brain through the activation of a receptor in the central nervous system.

Parkinson’s disease – Today, 1.5 million Americans suffer from Parkinson’s disease, and 60,000 new cases are diagnosed each year. A potential new medicine for Parkinson’s is a novel cell therapy that utilizes normal human cells attached to microcarriers to enhance brain levels of dopamine, the neurotransmitter deficient in Parkinson’s patients. The cells, retinal pigment epithelial cells, produce L-DOPA, a natural precursor of dopamine. The microcarriers, to which the cells are attached, enable long-term cell survival. The medicine is injected into the brain regions lacking dopamine using a surgical technique called sterotactic injection, which does not require general anesthesia.

Spinal Cord Injury – An estimated 225,000 to 228,000 people are alive today with spinal cord injuries, with an additional 11,000 new cases each year, according to the Spinal Cord Injury Information Network. One medicine in development is a monoclonal antibody that may have potential in the regeneration of corticospinal-tract fibers resulting from acute spinal injuries.

Stroke – About 700,000 Americans will have a stroke this year – that’s one every 45 seconds – according to the American Heart Association. A recombinant anticoagulant in development rapidly dissolves blood clots by directly degrading fibrin, a protein that provides the base for blood clots. In addition, it appears that its thrombolytic activity is localized to the site of delivery because it is rapidly deactivated by a protein in the blood as it moves into the general blood circulation.